Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy

NCT07704099 | Not Yet Recruiting Phase 2 FDA Drug

• 1 other identifier: KER-065-B201
• Study Type: interventional
• Target: 36
• Locations: 0 countries
• Sites: N/A

Brief Summary

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of KER-065 administered to adult and pediatric ambulatory and nonambulatory male participants with Duchenne Muscular Dystrophy (DMD) on stable background therapy.

Conditions

Duchenne Muscular Dystrophy

Keywords

Recombinant fusion protein; Muscle-wasting disease; Ambulatory function; Pharmacokinetics; Pharmacodynamics

Outcome Measures

Primary Outcomes (1)

• Number of participants with treatment-emergent adverse events (TEAEs and serious adverse events (SAEs)

  To evaluate the safety and tolerability of KER-065 in ambulatory and nonambulatory participants with DMD

  Up to approximately 3 years

Secondary Outcomes (9)

• KER-065 serum concentration by visit, as appropriate

  Up to Week 100

• Number and proportion of participants with treatment-emergent ADA (antidrug antibody) by visit

  Up to Week 100

• Change from baseline by visit in bone mineral density (BMD), fat mass, and lean body mass, as measured by dual- energy X-ray absorptiometry (DXA)

  Up to Week 96

• Change from baseline by visit in muscle volume and intramuscular fat by skeletal muscle MRI

  Up to Week 96

• Ambulatory: Change from baseline by visit in North Star Ambulatory Assessment (NSAA) total score

  Up to Week 96

Study Arms (3)

Cohort A1 (Late Ambulatory)

EXPERIMENTAL

Participants will receive stable corticosteroid (CS) along with KER-065.

Drug: KER-065

Cohort A2 (Late Ambulatory)

EXPERIMENTAL

Participants will receive stable CS, exon skipper along with KER-065.

Drug: KER-065

Cohort N1 (Nonambulatory)

EXPERIMENTAL

Participants will receive stable CS along with KER-065.

Drug: KER-065

Interventions

KER-065 DRUG

KER-065 will be administered subcutaneously (SC)

Cohort A1 (Late Ambulatory); Cohort A2 (Late Ambulatory); Cohort N1 (Nonambulatory)

Eligibility Criteria

• Age: 9 Years+
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of DMD, defined as the presence of phenotypic features at screening consistent with DMD AND documented mutation in the dystrophin gene consistent with the diagnosis of DMD using a clinically validated genetic test.
• Receiving a stable regimen of systemic CS (including, but not limited to, prednisone, prednisolone, deflazacort, or vamorolone) for at least 90 days before screening.
• Body weight of ≥ 25.0 kg.
• Ambulatory Participants Only (Cohort A1 and A2):
• Ambulatory, defined as able to walk independently without assistive devices.
• Able to TTR in \< 10 seconds.
• Has a NSAA score ≥ 15 points.
• Cohort 2 only: Documentation of a stable dose of an approved exon-skipping therapy.
• Nonambulatory Participants Only (Cohort N1):
• Nonambulatory, characterized as being unable to ambulate for a minimum of 3 months before first dose with onset of nonambulatory status AND a NSAA walk score of 0 and inability to perform the 10MWR.
• PUL v2.0 entry item score of 3 to 5, inclusive.

You may not qualify if:

• Clinical symptoms or signs of cardiomyopathy or heart failure.
• Exposure to any approved or investigational dystrophin restoration gene therapy product.
• Exposure to any approved or investigational dystrophin restoration product other than gene therapy (Except for exon-skipping therapy for Cohort A2).
• Exposure to any approved or investigational histone deacetylase inhibitor, antimyostatin therapy, therapy targeting transforming growth factor-beta ligands, or cell-based therapy.
• Use of any other pharmacological treatment, except for CS
• Treatment with immunosuppressant therapy (other than CS)
• History of fracture of the upper limb
• Nonambulatory Participants Only (Cohort N1):
• Elbow-flexion contractures \> 30° in both upper extremities.
• Forced vital capacity (FVC) of \< 50% or requirement for daytime or nocturnal ventilation, except for nocturnal non-invasive ventilation AND inability to perform consistent FVC measurements within ± 15% during paired testing.

Sponsors & Collaborators

• Keros Therapeutics, Inc.: lead

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Gina Weaver

CONTACT

267.799.3345; gweaver@kerostx.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 9, 2026
• First Posted: July 15, 2026
• Study Start (Estimated): September 14, 2026
• Primary Completion (Estimated): July 17, 2029
• Study Completion (Estimated): August 14, 2029
• Last Updated: July 15, 2026

Record last verified: 2026-07